Recruitment procedures for descriptive socio-economic studies in rare diseases. The BURQOL-RD project

Bibliography

• Taruscio D, Gentile AE, Evangelista T, et al. Centres of expertise and european reference networks: key issues in the field of rare diseases. The EUCERD Recommendations. Blood Transfus 2014;12(Suppl 3):s621-5
   PubMed Web of Science ®Google Scholar
• Communication from the Commission to the European Parliament, the Council, the European Economic and Social Committee and the Committee of the Regions” on Rare Diseases: Europes’ Challenges, (2009/C 151/02), 11 Nov 2008. Available from: http://ec.europa.eu/health/ph_threats/non_com/docs/rare_com_en.pdf [Last accessed 1 April 2015]
   Google Scholar
• Griggs RC, Batshaw M, Dunkle M, et al. Clinical research for rare disease: opportunities, challenges and solutions. Mol Genet Metab 2009;96(1):20-6
   PubMed Web of Science ®Google Scholar
• Kinder BW, Sherman AC, Young LR, et al. Predictors for clinical trial participation in the rare lung disease lymphangioleiomyomatosis. Respir Med 2010;104(4):578-83
   PubMed Web of Science ®Google Scholar
• Gupta S, Bayoumi AM, Faughnan ME. Rare lung disease research: strategies for improving identification and recruitment of research participants. Chest 2011;140(5):1123-9
   PubMedGoogle Scholar
• Carroll R, Antigua J, Taichman D, et al. Motivations of patients with pulmonary arterial hypertension to participate in randomized clinical trials. Clin Trials 2012;9(3):348-57
   Google Scholar
• De Ward SJ, Wilson A, Bausell H, Volz AS and Mooney K. Practical aspects of recruitment and retention in clinical trials of rare genetic diseases: the phenylketonuria (PKU) experience. J Genet Counsel 2014;23:20-8
   Google Scholar
• Johnson KJ, Mueller NL, Williams K, Gutmann DH. Evaluation of participant recruitment methods to a rare disease online registry. Am J Med Genet Part A 2014;164A:1686-94
   PubMed Web of Science ®Google Scholar
• Schumacher KR, Stringer KA, Donohue JE, et al. Social media methods for studying rare diseases. Pediatrics 2014.133;e1345-53. Available from: http://pediatrics.aappublications.org/content/133/5/e1345.full.html [Last accessed 15 April 2015]
   Google Scholar
• International rare diseases research consortium (IRDiRC). Available from: http://www.irdirc.org/ [Last accessed 15 April 2015]
   Google Scholar
• Global rare diseases patient registry and data repository (GRDR). Available from: http://www.grdr.info/ [Last accessed 1 March 2015]
   Google Scholar
• Caldwell PHY, Hamilton S, Tan A, Craig JC. Strategies for increasing recruitment to randomised controlled trials: systematic review. PLoS Med 2010;7(11):e1000368
   PubMed Web of Science ®Google Scholar
• Treweek S, Pitkethly M, Cook J, et al. Strategies to improve recruitment to randomised controlled trials. Cochrane Database Syst Rev 2010(4):MR000013
   Google Scholar
• Fayter D, McDaid C, Eastwood A. A systematic review highlights threats to validity in studies of barriers to cancer trial participation. J Clin Epidemiol 2007;60(10):990e1001
   Google Scholar
• Faurisson F. Survey of the delay in diagnosis for 8 rare diseases in Europe: EurordisCare2. European Organisation for Rare Diseases Web site. 2004. Available from: http://www.eurordis.org/sites/default/files/publications/Fact_Sheet_Eurordiscare2.pdf [Last accessed 1 May 2014]
   Google Scholar
• Linertová R, Serrano-Aguilar P, Posada-de-la-Paz M, López bastida J, et al. BURQOL-RD Research Group. Delphi approach to select rare diseases for a European representative survey. The BURQOL-RD study. Health Policy 2012;108(1):19-26
   Google Scholar
• French Ministry of Research. French national plan for rare diseases 2005-2008. Ensuring equity in the access to diagnosis, treatment and provision of care. EURORDIS. 2004.http://www.eurordis.org/IMG/pdf/EN_french_rare_disease_plan.pdf [Last accessed 15 April 2015]
   Google Scholar
• European Commission. Seventh Framework Program. Health.2010.2.4.4-2. ERA-Net on rare diseases (E-Rare-2). P7-ERANET-2010-RTD. Grant agreement no: 266608
   Google Scholar
• Westermann CJ, Rosina AF, De Vries V, de Coteau PA. The prevalence and manifestations of hereditary hemorrhagic telangiectasia in the Afro-Caribbean population of the Netherlands Antilles: a family screening. Am J Med Genet A 2003;116A(4):324-8
   PubMed Web of Science ®Google Scholar
• Ingelfinger JR, Drazen JM. POs and research on rare diseases. N Engl J Med 2011;364(17):1670-1
   Google Scholar
• Eurordis. The Voice of 12.000 Patients Experiences and expectations of rare disease patients on diagnosis and care in europe. Available from: www.eurordis.org 2009
   Google Scholar
• FEDER. Estudio sobre situación de necesidades sociosanitarias de las personas con enfermedades raras en España. Estudio ENSERio. Obra Social de Caja Madrid 2009
   Google Scholar
• Lauren Limb, Stephen Nutt and Alev Sen - December 2010 Experiences of Rare Diseases: An Insight from Patients and Families. Rare Diseases UK
   Google Scholar
• Rare Disease Impact Report: Insights from patients and the medical community. Shire 2013. Available from: http://www.shire.com/shireplc/dlibrary/documents/RareDiseaseImpactReportforWeb.pdf [Last accessed 23 March 2015]
   Google Scholar
• Bedgood R, Sadurski R, Schade RR. The use of the internet in data assimilation in rare diseases. Dig Dis Sci 2007;52(2):307-12
   PubMed Web of Science ®Google Scholar
• Fox S. Peer-to-peer Healthcare [Internet]. Pew Research Center. Retrieved from http://www.pewinternet.org/∼/media//Files/Reports/2011/Pew_P2PHealthcare_2011.pdf [Last accessed in April 2013]
   Google Scholar
• Hamilton RJ, Bowers BJ. Internet recruitment and e-mail interviews in qualitative studies. Qual Health Res 2006;16(6):821-35
   PubMed Web of Science ®Google Scholar
• Rhodes SD, Bowie DA, Hergenrather KC. Collecting behavioural data using the world wide web: considerations for researchers. J Epidemiol Community Health 2003;57(1):68-73
   PubMed Web of Science ®Google Scholar
• Abma TA. Patients as partners in a health research agenda setting. The feasibility of a participatory methodology. Evaluation & the Health Professions 2006;29(4):424-39
   PubMed Web of Science ®Google Scholar
• Oliver S, Clarke-Jones L, Rees R, et al. Involving consumers in research and development agenda setting for the NHS: developing an evidence-based approach. Health Technol Assess 2004;8(15):1-148
   PubMed Web of Science ®Google Scholar
• Whitstock MT. Seeking evidence from medical research consumers as part of the medical research process could improve the uptake of research evidence. J Eval Clin Pract 2003;9(2):213-24
   Google Scholar
• Serrano-Aguilar P, Trujillo-Martin MM, Ramos-Goñi JM, et al. Patient involvement in health research: A contribution to a systematic review on the effectiveness of treatments for degenerative ataxias. Soc Sci Med 2009;69:920-5
   PubMed Web of Science ®Google Scholar